Raise Your Voice
Your voice can help influence research funding budgets, the development of new therapies, clinical trial efforts and initiatives to improve the quality and cost of care for those affected by myotonic dystrophy (DM).
MDF’s advocacy program seeks to advocate for key initiatives that will drive Care and a Cure for DM while raising visibility of myotonic dystrophy and people living with DM with stakeholders in Congress, federal and state agencies, medical professionals and the media.
Federal funding for DM research ranks near the bottom of all federally funded disease research. Today, the federal government spends approximately $9 million dollars on DM research out of a total budget of just over $32 billion at the National Institutes of Health. This translates into approximately $11 per person per year.
Myotonic is currently urging Congress to add DM to the list of conditions eligible for research funding under the $2.1 billion Department of Defense Congressionally-Directed Medical Research Programs. This program was established by Congress in 1992 to seek novel and groundbreaking approaches to biomedical research. We are also pursuing a similar effort to increase DM-targeted funding at the National Institutes of Health (NIH), the largest US funder of medical and scientific research. Thanks to Myotonic efforts, myotonic dystrophy was eligible for CDMRP funding in FY 2018 and our researchers successfully competed for over $3M in new research funding! We also successfully advocated for eligibility in the FY 2019 budget, and have received notification that DM will be included in the 2020 federal program as well.
Myotonic is also advocating with regulatory agencies like the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) to share with them DM patient perspectives on the burden of living with this disease and patient perspectives on meaningful benefits of potential treatments. We have held a workshop on biomarkers and endpoints for clinical trials, and an all day meeting on clinical trial design and readiness for a multisystemic, variable and slow-progressing disease like DM. We will continue this work in fall 2016 with a Patient Focused Drug Development meeting with the FDA, which will develop patient insights and data that will be used in the review process of future therapies.
Please click the links below to learn more and get involved. Together we can accelerate research and care for myotonic dystrophy.
What is Grassroots Advocacy?
Grassroots advocacy is citizen participation in government. The key to successful advocacy is assembling people who share common goals and concerns. Advocacy is all about educating legislators about the opinions and views of their voting constituents.
Why should I become an advocate?
You have the most powerful tool available on Capitol Hill: your vote. YOU have the ability to hold your legislators accountable to you as their constituent. Without hearing from you, your legislator does not know what is important to you, or to the DM community as a whole. Your recommendation may be the deciding factor in a member's decision to vote for or against specific legislation. You can make a difference!
Can one person really influence Congress?
Absolutely! Many do not realize that Congress is a reactionary body. The voice of a constituency is what causes changes to the current conditions in government: when a citizenry assembles with a collective purpose and singular voice, the system will respond to it.
How a Bill Becomes a Law
- Idea for bill is generated
- Bill is written, sponsored & introduced
- Bill referred to committee for futher action
- Public hearings occur
- Expert testimony occurs
- Markup occurs
- Bill reported favorably, unfavorably, or tabled by committee
- Bill moves to House or Senate floor for debate & voting
- Approved bill moves to other chamber for same process
- Both chambers meet to reconcile bill differences
- Majority of both chambers must approve revisions
- Passed bill is sent to President to veto or sign into law!
Raise Your Voice
We need you to tell Congress how important our issues are to the DM community, and to create relationships with your members of Congress and their staff. You don't need to be in Washington to interact with them! Explore this section to learn ways that you can take action as an advocate right from your hometown. If you have any questions or need help with any of these action items, please contact Myotonic at 415-800-7777 or firstname.lastname@example.org.
Make a Phone Call
It will only take a minute, but it can mean so much to those living with DM! Tell them a little about your connection to DM, and offer to send them materials about the disease. If there is a particular issue that you would like to bring up (e.g. medical research, 21st Century Cures, etc.), mention it briefly. Be sure to identify yourself as a constituent by stating your address and city when speaking.
Phone numbers for your members of Congress can be found in the House or Senate directories. You can also call the US Capitol Switchboard at 202-234-3121.
Visit a District Office
A great way to familiarize your members of Congress with DM and your family's story is to give them a packet of information. To find your member of Congress' district office locations, please refer to the House of Representatives or Senate directories. We recommend including the following items in your informational packet:
Once you've dropped off the packet to your member of Congress' district office, let us know! We want to reach as many legislators as possible with these important messages.
Use the links below to find out more information that will help you become a successful advocate! If you have any questions or need help with any advocacy action items, please contact Myotonic at 415-800-7777, or email@example.com.
The barrage of government and procedural jargon is often confusing and off-putting for new advocates. Click here to view a glossary designed to help you learn some of the more commonly-used terms in the legislative process.
Myotonic Advocacy Webinars & Videos
Myotonic regularly presents advocacy webinars explaining our current issues and campaigns, and provides advocacy training's at our annual conference.
- If you haven't already done so, register to vote to make your voice heard on important issues!
- If you need to find a phone number for a member of Congress, call the US Capitol Switchboard at (202) 234-3121 or visit the House of Representatives or Senate directories.
- You can find information on bills and other legislation via THOMAS, the Library of Congress' online portal.
- Visit Project Vote Smart to learn how your Representative or Senators voted on a specific issue.
Sign-on Letters & Comments
Myotonic partners with many different patient advocacy organizations to fulfill our legislative priorities. To that end, we have joined these organizations in calling on Congress, the NIH, the FDA, and the administration to sign onto letters either supporting or offering additional commentary on proposed legislation. These comments and letters are available for download below.
- US Congress: Opposing the Repeal of the Genetic Information Nondiscrimination Act (4/21/15)
- House Committee on Energy & Commerce: Permanent Reauthorization of the Rare Pediatric Disease PRV Program (4/13/15)
- House Committee on Energy & Commerce: Access to 21st Century Cures for Individuals with Rare Diseases (3/17/15)
- House Committee on Ways & Means: Ensuring Access to Clinical Trials Act (3/12/2015)
- Senate Committee on Finance: Ensuring Access to Clinical Trials Act (3/12/2015)
- Muscular Dystrophy Coordinating Committee: Comments on MDCC Action Plan (3/10/2015)
- House Committee on Energy & Commerce: Patient-Focused Impact Assessment Act (1/22/2015)
- Secretary Burwell/Department of Health & Human Services: Draft Guidance on Disclosing Reasonably Foreseeable Risks in Research Evaluating Standards of Care (1/22/2015)
- Secretary Burwell/Department of Health & Human Services: Extension for Draft Guidance on Disclosing Reasonably Foreseeable Risks in Research Evaluating Standards of Care (11/18/2014)
- House Committee on Energy & Commerce: Support the MD-CARE Amendments (6/13/2014)
- Rep. Michael Burgess & Rep. Eliot Engel: Support the MD-CARE Amendments (6/13/2014)
- House Committees on Finance and Ways & Means: Save the Orphan Drug Tax Credit (3/10/2014)